Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
ATTRv amyloidosis
RNA interference
hATTR amyloidosis
patisiran
vutrisiran
Journal
Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
ISSN: 1744-2818
Titre abrégé: Amyloid
Pays: England
ID NLM: 9433802
Informations de publication
Date de publication:
Mar 2023
Mar 2023
Historique:
pubmed:
26
7
2022
medline:
7
3
2023
entrez:
25
7
2022
Statut:
ppublish
Résumé
The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy. HELIOS-A was a phase 3, global, open-label study comparing the efficacy and safety of vutrisiran with an external placebo group (APOLLO study). Patients were randomized 3:1 to subcutaneous vutrisiran 25 mg every 3 months (Q3M) or intravenous patisiran 0.3 mg/kg every 3 weeks (Q3W) for 18 months. HELIOS-A enrolled 164 patients (vutrisiran, Vutrisiran significantly improved multiple disease-relevant outcomes for ATTRv amyloidosis versus external placebo, with an acceptable safety profile. NCT03759379.
Sections du résumé
BACKGROUND
UNASSIGNED
The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis with polyneuropathy.
METHODS
UNASSIGNED
HELIOS-A was a phase 3, global, open-label study comparing the efficacy and safety of vutrisiran with an external placebo group (APOLLO study). Patients were randomized 3:1 to subcutaneous vutrisiran 25 mg every 3 months (Q3M) or intravenous patisiran 0.3 mg/kg every 3 weeks (Q3W) for 18 months.
RESULTS
UNASSIGNED
HELIOS-A enrolled 164 patients (vutrisiran,
CONCLUSIONS
UNASSIGNED
Vutrisiran significantly improved multiple disease-relevant outcomes for ATTRv amyloidosis versus external placebo, with an acceptable safety profile.
CLINICALTRIALS.GOV
UNASSIGNED
NCT03759379.
Identifiants
pubmed: 35875890
doi: 10.1080/13506129.2022.2091985
doi:
Substances chimiques
Prealbumin
0
Banques de données
ClinicalTrials.gov
['NCT03759379']
Types de publication
Randomized Controlled Trial
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1-9Investigateurs
J Wixner
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R Backlund
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